Adenine base editing

1. Adenine base editing extends lifespan in mice.

   “An adenine base editor (ABE7.10max) corrected the point mutation and produced significant improvement in HGPS mouse lifespan, vascular smooth muscle cell density, and adventitial fibrosis.”

   Reviewin HGPS mice (cited background)

   Adenine base editing rescues pathogenic phenotypes in tissue engineered vascular model of Hutchinson-Gilford progeria syndrome

   APL bioengineering·26 Feb 2025

2. Adenine base editing extends lifespan in mice.

   “Systemic delivery of ABE components containing ABEmax-VRQR-SpCas9 and single-guide RNA by adeno-associated virus serotype 9 in these mice restored cardiac function as assessed by echocardiography and extended life span.”

   Animalsin Rbm20 R636Q/R636Q mice

   Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy

   Science translational medicine·23 Nov 2022

1. Adenine base editing improves smooth muscle cell proliferation in human cells.

   “When TEBVs were prepared with at least 50% edited smooth muscle cells, viSMC proliferation and myosin heavy chain levels significantly improved.”

   Cellswhen TEBVs prepared with at least 50% edited smooth muscle cellsat least 50% edited smooth muscle cells

   Adenine base editing rescues pathogenic phenotypes in tissue engineered vascular model of Hutchinson-Gilford progeria syndrome

   APL bioengineering·26 Feb 2025

1. Adenine base editing improves vasodilation in human cells.

   “Normal vasodilation and viSMC density were restored in TEBVs made with edited cells.”

   Cellsin TEBVs made with edited cells

   Adenine base editing rescues pathogenic phenotypes in tissue engineered vascular model of Hutchinson-Gilford progeria syndrome

   APL bioengineering·26 Feb 2025

1. Adenine base editing inhibits progerin in human cells.

   “Endothelial cells (viECs) and smooth muscle cells (viSMCs) obtained by differentiation of edited HGPS iPSCs did not express progerin”

   Cellsin differentiated HGPS iPSCs

   Adenine base editing rescues pathogenic phenotypes in tissue engineered vascular model of Hutchinson-Gilford progeria syndrome

   APL bioengineering·26 Feb 2025

More on Progerin

1. Adenine base editing decreases ribonucleoprotein granules in human cells.

   “normalized alternative splicing of cardiac genes, restored nuclear localization of RBM20, and eliminated RNP granule formation.”

   Cellsin RBM20 R634Q human iPSC-derived cardiomyocytes

   Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy

   Science translational medicine·23 Nov 2022

1. Adenine base editing improves cardiac function in mice.

   “Systemic delivery of ABE components containing ABEmax-VRQR-SpCas9 and single-guide RNA by adeno-associated virus serotype 9 in these mice restored cardiac function as assessed by echocardiography”

   Animalsin Rbm20 R636Q/R636Q miceheart

   Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy

   Science translational medicine·23 Nov 2022